Titolo: National Center for Gene Therapy and Drugs based on RNA Technology
Dati identificativi pnrr  Codice identificativo MUR CN00000041 (PNRR), Missione 4, Componente 2
Data di inizio: 1 novembre 2022
Data di fine: 31 ottobre 2025
Durata: 3 anni
Abstract:
The proposed project aims at treating neurodegenerative diseases (NDs) like Alzheimer's Disease (AD), Parkinson’s Disease (PD) and in polyglutamine diseases, including Huntington's disease (HD), spinobulbar muscular atrophy (SBMA), and dominant forms of spinocerebellar ataxia (SCA).
We performed in vitro and preclinical studies in rodent models of NDs and developed three therapeutic approaches (“Verticals”). Crucially, these approaches are highly complementary and can be used for multiple diseases.
We have also developed tools (“Horizontals”) that are instrumental to consolidate our findings and take them to a clinical setting.

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